CLINICAL TRIALS PROFILE FOR NIPENT

505(b)(2) Clinical Trials for Nipent

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	California Institute for Regenerative Medicine (CIRM)	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	City of Hope Medical Center	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.

All Clinical Trials for Nipent

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00038025 ↗	A Study Of Deoxycoformycin(DCF)/Pentostatin In Lymphoid Malignancies	Completed	M.D. Anderson Cancer Center	Phase 2	1994-09-06	The purpose of this study is to determine the side effects and antitumor response of patients with lymphoid malignancies to Deoxycoformycin (DCF)/Pentostatin.
NCT00045305 ↗	Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes	Completed	National Cancer Institute (NCI)	Phase 2	2005-05-01	RATIONALE: Photopheresis treats the patient's blood with drugs and ultraviolet light outside the body and kills the white blood cells. Giving photopheresis, pentostatin, and radiation therapy before a donor bone marrow or stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving pentostatin before transplant and cyclosporine or mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving pentostatin together with photopheresis and total-body irradiation work before donor bone marrow transplant in treating patients with myelodysplastic syndromes.
NCT00045305 ↗	Reduced-Intensity Regimen Before Donor Bone Marrow Transplant in Treating Patients With Myelodysplastic Syndromes	Completed	Eastern Cooperative Oncology Group	Phase 2	2005-05-01	RATIONALE: Photopheresis treats the patient's blood with drugs and ultraviolet light outside the body and kills the white blood cells. Giving photopheresis, pentostatin, and radiation therapy before a donor bone marrow or stem cell transplant helps stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune system and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving pentostatin before transplant and cyclosporine or mycophenolate mofetil after transplant may stop this from happening. PURPOSE: This phase II trial is studying how well giving pentostatin together with photopheresis and total-body irradiation work before donor bone marrow transplant in treating patients with myelodysplastic syndromes.

Clinical Trial Conditions for Nipent

Condition Name

Condition Name for Nipent
Intervention	Trials
Chronic Lymphocytic Leukemia	5
Leukemia	4
Lymphoma	4

Condition MeSH

Condition MeSH for Nipent
Intervention	Trials
Leukemia	9
Leukemia, Lymphoid	7
Leukemia, Lymphocytic, Chronic, B-Cell	7

Clinical Trial Locations for Nipent

Trials by Country

Trials by Country for Nipent
Location	Trials
United States	85
Italy	1

Trials by US State

Trials by US State for Nipent
Location	Trials
Florida	6
Texas	6
Maryland	6
Ohio	4
Minnesota	4

Clinical Trial Progress for Nipent

Clinical Trial Phase

Clinical Trial Phase for Nipent
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	1
Phase 2	11

Clinical Trial Status

Clinical Trial Status for Nipent
Clinical Trial Phase	Trials
Completed	12
Terminated	2
Not yet recruiting	1

Clinical Trial Sponsors for Nipent

Sponsor Name

Sponsor Name for Nipent
Sponsor	Trials
National Cancer Institute (NCI)	9
Astex Pharmaceuticals	5
Astex Pharmaceuticals, Inc.	5

Sponsor Type

Sponsor Type for Nipent
Sponsor	Trials
Other	17
Industry	16
NIH	10

NIPENT: Clinical Trials Update, Market Analysis, and Projections

Overview of NIPENT

NIPENT, also known as pentostatin, is a potent inhibitor of the enzyme adenosine deaminase and is used as a single-agent treatment for both untreated and alpha-interferon-refractory hairy cell leukemia patients with active disease. It is supplied as a sterile, apyrogenic, lyophilized powder in single-dose vials for intravenous administration[2].

Mechanism of Action and Clinical Use

Pentostatin, or 2’-deoxycoformycin (DCF), is isolated from fermentation cultures of Streptomyces antibioticus. It works by inhibiting adenosine deaminase, an enzyme crucial for the breakdown of certain nucleosides. This inhibition leads to the accumulation of toxic metabolites that interfere with DNA synthesis, particularly affecting lymphoid cells, which are often involved in hairy cell leukemia[2].

Clinical Trials and Development Activities

While NIPENT itself is not currently in new clinical trials, the landscape of treatments for hairy cell leukemia is evolving. Here are some key points related to clinical trials in the broader context:

• Historical Trials: NIPENT has been through extensive clinical trials in the past, which have established its efficacy and safety profile for the treatment of hairy cell leukemia.
• Current Focus: The focus has shifted towards other emerging therapies and treatments for related conditions. For instance, new clinical trials are being conducted for other autoimmune and hematological diseases, which might indirectly influence the market for NIPENT[1][3].

Market Analysis

Market Size and Forecast

The global hairy cell leukemia treatment market, which includes NIPENT, is estimated to grow at a CAGR of 5.4% from 2024 to 2030. The market size was valued at USD 102.1 million in 2023 and is expected to increase significantly over the forecast period[5].

Regional Market

The market for NIPENT is analyzed across several major regions, including the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. The report provides historical and forecasted sales data for these regions, helping in understanding the market dynamics and potential growth areas[2].

Competitive Landscape

NIPENT faces competition from other approved products for hairy cell leukemia. Companies such as Pfizer Inc, F. Hoffmann La Roche AG, Janssen Global Services, and others are key players in this market. The launch of late-stage emerging therapies is expected to significantly impact the market, potentially altering the competitive landscape[5].

SWOT Analysis

• Strengths: Established safety and efficacy profile, long-standing presence in the market.
• Weaknesses: Potential competition from new therapies, limited scope of treatment to hairy cell leukemia.
• Opportunities: Growing healthcare spending, increasing prevalence of chronic diseases.
• Threats: Emergence of new treatments, regulatory changes, and market competition[2].

Regulatory Milestones and Development Activities

NIPENT has already received regulatory approvals for its use in treating hairy cell leukemia. Here are some key regulatory and development points:

• Regulatory Approvals: NIPENT is approved for use in several countries, including the United States, EU5, and Japan.
• Development Activities: While NIPENT itself is not in new development stages, the company and other stakeholders are involved in assessing challenges and seeking opportunities that could influence its market position. This includes monitoring emerging therapies and their potential impact on the market[2].

Market Projections

Sales Forecast

The sales data for NIPENT from 2017 to 2030 indicates a steady market presence. However, the market is expected to evolve due to the introduction of new therapies and increasing healthcare spending. The report provides detailed historical and forecasted sales data to support decision-making processes regarding therapeutic portfolios[2].

Impact of Emerging Therapies

The market for NIPENT will be influenced by the launch of late-stage emerging therapies. These new treatments are focused on novel approaches to treat or improve disease conditions, which could potentially alter the market dynamics and competition for NIPENT[2].

Global Market Trends

The global clinical trials market, which includes trials for various diseases, is projected to grow significantly. This growth is driven by the increasing prevalence of chronic diseases and the rising number of clinical trials. While NIPENT is not directly involved in these new trials, the overall trend indicates a growing demand for innovative treatments, which could impact its market position[3].

Key Takeaways

• Established Treatment: NIPENT is an established treatment for hairy cell leukemia with a proven safety and efficacy profile.
• Market Growth: The market for hairy cell leukemia treatments is expected to grow at a CAGR of 5.4% from 2024 to 2030.
• Competition: NIPENT faces competition from other approved products and emerging therapies.
• Regulatory Stability: NIPENT has received regulatory approvals in several countries.
• Market Evolution: The market is expected to evolve with the introduction of new therapies and increasing healthcare spending.

FAQs

What is NIPENT used for?

NIPENT is used as a single-agent treatment for both untreated and alpha-interferon-refractory hairy cell leukemia patients with active disease.

What is the mechanism of action of NIPENT?

NIPENT works by inhibiting the enzyme adenosine deaminase, leading to the accumulation of toxic metabolites that interfere with DNA synthesis, particularly affecting lymphoid cells.

What is the current market size and forecast for NIPENT?

The global hairy cell leukemia treatment market, which includes NIPENT, is estimated to grow at a CAGR of 5.4% from 2024 to 2030, with a market size valued at USD 102.1 million in 2023.

Who are the key competitors in the market for NIPENT?

Key competitors include Pfizer Inc, F. Hoffmann La Roche AG, Janssen Global Services, and others.

How will emerging therapies impact the market for NIPENT?

Emerging therapies are expected to significantly impact the market, potentially altering the competitive landscape and influencing the market position of NIPENT.

Sources

1. Nkarta Announces IND Clearance of Investigator-Sponsored Trial in Myasthenia Gravis and Opening of Enrollment for Ntrust-2. Nkarta, Inc.
2. Nipent - Drug Insight and Market Forecast - 2030. ASDReports.
3. Clinical Trials Market SIZE, SHARE | GROWTH REPORT [2032]. Fortune Business Insights.
4. Phase 3 Clinical Trials With Primary Completion Dates in January 2025. FDA Tracker.
5. Hairy Cell Leukemia Treatment Market Size Report, 2030. Grand View Research.